Gathering real-world compassionate data to expand eligibility for elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with N1303K or other rare CFTR variants: a viewpoint.

Competing Interests: Conflict of interest: P-R. Burgel reports support for the present manuscript from Association Vaincre la Mucoviscidose, Société Française de la Mucoviscidose and Filière Maladie Rare Muco CFTR; in addition, P-R. Burgel reports grants from Vertex Pharmaceuticals and GSK, consulting fees from AstraZeneca, Chiesi, GSK, Insmed, Vertex, Viatris and Zambon, and travel support AstraZeneca and Chiesi, outside the submitted work. I. Sermet-Gaudelus reports support for the present manuscript from Vertex Therapeutics and Tavanta; in addition, I. Sermet-Gaudelus reports grants from Vertex Therapeutics and Tavanta, outside the submitted work. I. Durieu and J. Languepin report travel support from Mylan, outside the submitted work. A. Guillaumot reports travel support from Asten, Boehringer Ingelheim, GSK, LFB, CSL Behring, Roche and Menarini, outside the submitted work. C. Audousset reports travel support from Zambon and Viatris, and advisory board participation from Vertex Pharmaceuticals, outside the submitted work. R. Chiron reports travel support from ECFC, and advisory board participation with Zambon and Vertex, outside the submitted work. L. Weiss reports travel support from Viatris, and advisory board participation from Vertex, outside the submitted work. I. Fajac reports grants from AbbVie, Bayer, Boehringer Ingelheim, Insmed, GSK, Vertex Pharmaceuticals and Zambon, consulting fees from AbbVie, Boehringer Ingelheim, Kither Biotech and Vertex Pharmaceuticals, and a leadership role with the European Cystic Fibrosis Society, outside the submitted work. C. Martin reports lecture honoraria from Chiesi, AstraZeneca, Boehringer and GSL, and travel support from Chiesi and Boehringer, outside the submitted work. The remaining authors have no potential conflicts of interest to disclose.